PI-01: GRNDAD AND COUSIN: INVESTIGATOR-INITIATED COLLABORATIONS TO IMPROVE CARE IN SICKLE CELL DISEASE
Authors: LITTLE J., MANWANI D., KANTER J., DESAI P., GARDNER K., HOWARD J., LANZKRON S.
Journal: HemaSphere (2022)

Purpose: The Globin Research Network for Data and Discovery, GRNDaD (2016), registry and its recently initiated partner, Collaborating to Understand Sickle Cell Disease Internationally, CoUSIn (2021), were developed by providers at sickle cell centers in order to collect prospective data on the modern cohort of people living with SCD. The goal of these registries is to have the tools to describe the natural history of the disease and ensure the provision of guideline-based care. Neither foundations nor government-supported registries have, to date, been able to supply a comprehensive prospective detailed and patient-centered registry like GRNDaD or CoUSIn. Materials and methods: GRNDaD, comprising over 300 individual data elements, collects data from 12 sites at present, using de-identified data, and relying on a central IRB and annual updates (Table 1, N-1319, Fall 2019). The prospective REDCap database has been flexibly amenable to the addition of more highly detailed data sheets, encompassing queries on end stage renal disease, pregnancy, and thrombophilia. Results: To date, 12 sites in the US are fully functional, with 8 additional sites on-boarding. GRNDaD serves as the companion registry for the National Alliance of Sickle Cell Centers, NASCC, and is expected to grow briskly in the next 24-36 months. Most patients have sickle cell anemia (HbSS or HbSb0, Table 1), many have contributed patient reported outcome (PRO) surveys, and half of all entrants are adolescent/young adults (percent of total, Figure 1). As of February 2022, 2160 people with SCD have consented for data entry. There is a robust range of treatments in GRNDaD; of 2160 subjects, 53% of subjects with HgbSS or SB0thalassemia were on hydroxyurea, 12% of all subjects were on chronic transfusions, 2.4% were on L-glutamine, 2.2%were on crizanlizumab, and 2.0% were on Voxelotor. We estimate that >100 patients are likely to be added each year for recently-approved novel therapies Crizanlizumab and Voxelotor. Of note, the older adult with SCA had a depressed reticulocyte count and a trend towards a higher creatinine (not shown). Finally, the GRNDaD data dictionary has been shared with investigators in the UK, who have developed an identical parallel database, CoUSIn, which will serve as a platform for comparisons going forward of prospectively collected data between SCD populations that are cared for in 2 entirely distinct settings. Conclusion: Multi-site prospective registries designed to better understand the contemporary person living with SCD are crucially important, for tracking, and analysis of guideline-based care delivery in this disease. With funding from several sources, these registries will continue to grow and have plans for sustainability well into the future.Shown are sites, genotype distribution, and patient reported outcome survey attainment in GRNDaD, as of Fall 2019.Shown is the age distribution for the contemporary SCD population (%), in GRNDaD, as of February 2022. S. LANZKRON declares a conflict of interest: Consultancy, Expert: Pfizer, Novartis, BlueBird Bio, Magenta Research support/Scientific studies: Imara, GBT, Shire, Novartis, CSL Behring Stock shareholder: Pfizer, Teva